A Phase 2, Multicenter, Open-Label Study to Assess PK/PD of SEG101 (Crizanlizumab), With or Without Hydroxyurea/Hydroxycarbamide, in Sickle Cell Patients With Vaso-Occlusive Crisis

Locations

12 United States sites

Age

16 to 70 Years

Phase

Phase 2

Study type

Interventional

Gender

All

Interventions

Drug

Compensation

Unknown

About the study

The purpose of the CSEG101A2202 study was to characterize the PK and PD of
SEG101/crizanlizumab at 5 mg/kg and to evaluate the safety and efficacy of
SEG101/crizanlizumab in SCD patients.

Study CSEG101A2202 was designed as a Phase II, multicenter, open-label study. The first 45
patients (to identify 27 evaluable patients) were enrolled to the treatment group
crizanlizumab 5.0 mg/kg to complete full PK/PD sampling at week 1 and week 15. In all
patients, trough PK/PD samples was collected prior to each dose. In addition, throughout the
study (and when possible), all patients had blood drawn for serum to assess PK and PD drawn
at times of onset and resolution of each VOC event, fever, or infection. Once the up to 45
patients were enrolled, 12 additional patients were enrolled to the exploratory treatment
group and begin at 7.5 mg/kg of crizanlizumab.

participation requirements

– Male and non-pregnant female patients 16-70 years of age (inclusive)

– Confirmed diagnosis of sickle cell disease by hemoglobin electrophoresis or
high-performance liquid chromatography (HPLC) [performed locally]. All sickle cell
disease genotypes are eligible.

– Experienced at least 1 VOC within the preceding 12 months prior to Screening, as
determined by medical history.

– If receiving HU/HC or erythropoietin stimulating agent, must have been receiving the
drug for at least 6 months prior to Screening

– Hemoglobin ≥4.0 g/dL. Absolute neutrophil count ≥1.0 x 109/L and platelet count ≥75 x
109/L

– Adequate renal and hepatic function as defined:

– GFR ≥45 mL/min/1.73 m2 calculated by CKD-EPI

– ALT ≤3 x ULN

– Direct (conjugated) bilirubin ≤2 x ULN

– ECOG performance status ≤2

– Written informed consent (or assent/ parental consent for minor subjects) prior to any
screening procedures

participation restrictions

– History of stem cell transplant.

– Acute VOC ending 7 days prior to first dosing

– Ongoing hospitalization prior to Screening

– Received blood products within 30 days to first dosing

– Participating in a chronic transfusion program (pre-planned series of transfusions for
prophylactic purposes)

– History of severe hypersensitivity reactions to other monoclonal antibodies

– Received a monoclonal antibody or immunoglobulin -based agent within 1 year of
Screening, or has documented immunogenicity to a prior biologic.

– Received active treatment on another investigational trial within 30 days (or 5
half-lives of that agent, whichever is greater) prior to Screening

– Significant active infection or immune deficiency (including chronic use of
immunosuppressive drugs)

– Resting QTcF ≥470 msec at pretreatment (baseline) or other cardiac or cardiac
repolarization abnormality

Locations

  • Orange City, Florida, United States, Mid Florida Hematology and Oncology Center, 32763
  • Tampa, Florida, United States, Tampa General Hospital, 33606
  • Atlanta, Georgia, United States, Childrens Healthcare of Atlanta, 30342
  • Augusta, Georgia, United States, Augusta University Georgia Cancer Center Pharmacy Patient Treatment, 30912
  • Baltimore, Maryland, United States, University of Maryland Medical Center, 21201
  • Bronx, New York, United States, Children's Hospital at Montefiore, 10467
  • Durham, North Carolina, United States, Duke University Medical Center Patient Treatment, 27710
  • Greenville, North Carolina, United States, East Carolina University East Carolina University, 27858
  • Philadelphia, Pennsylvania, United States, Children s Hospital of Philadelphia Patient Treatment, 19104-4399
  • Charleston, South Carolina, United States, Medical University of South Carolina Medical Univ of SC, 29425
  • Columbia, South Carolina, United States, M Francisco Gonzalez MD PA, 29203
  • Rock Hill, South Carolina, United States, Carolina Blood and Cancer Care of South Carolina, 29732